Table 3.
Prioritized PICO questions and outcomes
| Final question(s) in PICO format | Selected outcomes | |
|---|---|---|
| Adult ITP | ||
| Should adults with newly diagnosed ITP and a platelet count <30 × 109/L who are asymptomatic be treated with corticosteroids or observation? | Major bleeding | Remission |
| Should adults with newly diagnosed ITP and a platelet count ≥30 × 109/L who are asymptomatic be treated with corticosteroids or observation? | Overall health-related quality of life | Mortality |
| Response within 7 d | ||
| Should adults with newly diagnosed ITP and a platelet count <20 × 109/L and no or mild bleeding be treated as an outpatient or be admitted to the hospital? | Major bleeding | |
| Should adults with newly diagnosed ITP and a platelet count ≥20 × 109/L and no or mild bleeding be treated as an outpatient or be admitted to the hospital? | Mortality | |
| Should adults with newly diagnosed ITP receive a shorter (≤ 8 wk) or prolonged course (including treatment and taper) of corticosteroids for initial therapy? | Durable response | Infection |
| Major bleeding | Mortality | |
| Remission | ||
| Should adults with newly diagnosed ITP be treated with prednisone (0.5-2.0 mg/kg per day) or dexamethasone (40 mg/day × 4 d) as the type of corticosteroid for initial therapy? | Durable response | Major bleeding |
| Overall HRQoL | Remission | |
| Response within 7 d | Response within 1 mo | |
| Should adults with newly diagnosed ITP be treated with rituximab and corticosteroids or corticosteroids alone for initial therapy? | Durable response | Infection |
| Major bleeding | Mortality | |
| Overall HRQoL | Remission | |
| Response within 1 mo | ||
| If an adult with ITP is corticosteroid dependent or unresponsive to corticosteroids and is going to be treated with a TPO-RA, should the patient receive eltrombopag or romiplostim? | Durable response | Major Bleeding |
| Overall HRQoL | Remission | |
| Response within 1 mo | Thrombosis | |
| Reduction or discontinuation of corticosteroids | ||
| Should adults with ITP lasting ≥3 mo who are corticosteroid dependent or have no response to corticosteroids undergo splenectomy or be treated with TPO-RAs? | Durable response | Infection |
| Major bleeding | Operative complications | |
| Overall HRQoL | Remission | |
| Response within one month | Thrombosis | |
| Reduction/discontinuation of corticosteroids | ||
| Should adults with ITP lasting ≥3 mo who are corticosteroid dependent or have no response to corticosteroids be treated with rituximab or TPO-RAs? | Durable response | Infection |
| Major bleeding | Thrombosis | |
| Overall HRQoL | Remission | |
| Response within one month | ||
| Reduction/discontinuation of corticosteroids | ||
| Should adults with ITP lasting ≥3 mo who are corticosteroid dependent or have no response to corticosteroids undergo splenectomy or be treated with rituximab? | Durable response | Infection |
| Major bleeding | Operative complications | |
| Overall HRQoL | Remission | |
| Response within one month | Thrombosis | |
| Reduction/discontinuation of corticosteroids | ||
| Pediatric ITP | ||
| Should children with newly diagnosed ITP and a platelet count <20 × 109/L be treated as an outpatient or be admitted to the hospital? | Major bleeding | Mortality |
| Should children with newly diagnosed ITP and a platelet count ≥20 × 109/L be treated as an outpatient or be admitted to the hospital? | Overall HRQoL | |
| Should children with newly diagnosed ITP and no or minor bleeding be treated with observation or corticosteroids for initial therapy? | Durable response | Major bleeding |
| Overall HRQoL | Mortality | |
| Remission | ||
| Should children with newly diagnosed ITP and no or minor bleeding be treated with observation or IVIg? | Durable response | Major Bleeding |
| Overall HRQoL | Mortality | |
| Remission | ||
| Should children with newly diagnosed ITP and no or minor bleeding be treated with observation or anti-D immunoglobulin for initial therapy? | Durable response | Hemolysis |
| Major bleeding | Mortality | |
| Overall HRQoL | Remission | |
| Should children with newly diagnosed ITP who as determined above require treatment, be treated with anti-D immunoglobulin or corticosteroids for initial therapy? | Durable response | Hemolysis |
| Major bleeding | Mortality | |
| Overall HRQoL | Remission | |
| Should children with newly diagnosed ITP who as determined above require drug therapy receive IVIg or anti-D immunoglobulin for initial therapy? | Durable response | Hemolysis |
| Major bleeding | Mortality | |
| Overall HRQoL | Remission | |
| Should children with newly diagnosed ITP who as determined above require drug therapy receive a course of corticosteroids longer or shorter than 7 d? | Durable response | Infection |
| Major bleeding | Mood or mental changes | |
| Overall HRQoL | Mortality | |
| Remission | ||
| Should children with newly diagnosed ITP who as determined above require drug therapy receive dexamethasone (0.6 mg/kg per day for 4 d every 4 wk) or prednisone (2-4 mg/kg per day × 5-7 d) as the type of corticosteroid? | Durable response | Major bleeding |
| Mortality | Remission | |
| What is the best treatment of children who are unresponsive to first-line treatment? That is, what are the risks and benefits to various treatments: splenectomy, rituximab, and TPO-RAs? | Durable response | Infection |
| Major bleeding | Thrombosis | |
| Overall HRQoL | Remission | |
| Response within 1 month | ||
| Reduction/discontinuation of corticosteroids | ||
PICO, population, intervention, comparator, and outcome.